Abu Dhabi Health Authority has reached a significant milestone by providing an innovative gene therapy treatment for Duchenne Muscular Dystrophy (DMD) for the first time.
The Authority announced that this groundbreaking treatment is available at Sheikh Khalifa Medical City.
This development underscores Abu Dhabi’s position as a global leader in healthcare and life sciences.
A specialised medical team, led by a paediatric neurology consultant at Sheikh Khalifa Medical City, in collaboration with a multidisciplinary team from the Department of Health’s Research and Innovation Centre, successfully administered the treatment for this rare disease to an Emirati patient in March.
Duchenne Muscular Dystrophy is a neuromuscular disorder leading to a progressive decline in muscle function. Notably, in the past, this treatment was only available in the United States.
The team utilised a single-dose injection of “Delandistrogene moxeparvovec,” which has proven effective in addressing the disorder by delivering functional dystrophin genes to the patient’s cells.
This treatment guides the cells to produce the vital dystrophin protein, enhancing muscle strength and improving the patient’s quality of life.
The gene therapy is specifically aimed at children aged four to five with a confirmed mutation in the DMD gene. However, it is not suitable for those with deletions in exons 8 and 9 of the DMD gene.
Moreover, Dr Noura Khamis Al Ghaithi, Undersecretary of the Department of Health – Abu Dhabi, stated, “This achievement highlights Abu Dhabi’s crucial role in adopting innovative healthcare solutions that provide exceptional, comprehensive care for all community members.
Abu Dhabi continues its efforts to offer advanced preventive, diagnostic, and therapeutic capabilities to local and international communities, reinforcing its status as a leading destination for healthcare and medical tourism.”
Mohamed El Shaarawy, General Manager of Roche UAE, expressed pride in Roche’s collaboration with the Department of Health – Abu Dhabi, emphasising their commitment to innovation and patient-centred care. He highlighted the importance of facilitating access to high-quality healthcare solutions, transcending traditional pharmaceutical boundaries, and exploring opportunities for research, education, and community awareness.
Dr Asma Al Mannaei, Executive Director of the Research and Innovation Centre at the Department of Health – Abu Dhabi, remarked, “The availability of Delandistrogene moxeparvovec enhances patient outcomes and creates valuable research opportunities, fostering a better understanding and development of therapeutic interventions, thereby enriching medical knowledge both regionally and internationally.”
She further noted the challenges in estimating the prevalence of DMD due to irregular genetic testing. However, the Emirati Genome Programme offers a promising opportunity to establish a national genetic registry, which could transform the landscape of genetic research and treatment.
Dr Omar Ismail, Consultant Paediatric Neurologist and Head of the Paediatric Neurology Department at Sheikh Khalifa Medical City, expressed his excitement about the first-time use of Delandistrogene moxeparvovec in Abu Dhabi, marking a new chapter in the management of Duchenne Muscular Dystrophy.
DMD results from a defect in the dystrophin protein, crucial for maintaining muscle cell integrity. The absence of functional dystrophin leads to the breakdown of muscle fibres, causing progressive muscle weakness and deterioration.
This weakness can result in life-threatening complications, such as heart or respiratory failure. Research indicates that DMD predominantly affects males, with an estimated incidence of one in 3,500 to 5,000 male births worldwide.
Moreover, the average lifespan of those affected is around 30 years, often characterised by the loss of walking ability and upper limb, lung, and heart function.
